Use of Real-World Data and Real-World Evidence in Rare Disease Drug Development: A Statistical Perspective
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arXiv
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| Autori principali: | , , , , , , , , , , , , , , , |
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| Natura: | Preprint |
| Pubblicazione: |
2024
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| _version_ | 1866909343026249728 |
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| author | Chen, Jie Gruber, Susan Lee, Hana Chu, Haitao Lee, Shiowjen Tian, Haijun Wang, Yan He, Weili Jemielita, Thomas Song, Yang Tamura, Roy Tian, Lu Zhao, Yihua Chen, Yong van der Laan, Mark Nie, Lei |
| author_facet | Chen, Jie Gruber, Susan Lee, Hana Chu, Haitao Lee, Shiowjen Tian, Haijun Wang, Yan He, Weili Jemielita, Thomas Song, Yang Tamura, Roy Tian, Lu Zhao, Yihua Chen, Yong van der Laan, Mark Nie, Lei |
| contents | Real-world data (RWD) and real-world evidence (RWE) have been increasingly used in medical product development and regulatory decision-making, especially for rare diseases. After outlining the challenges and possible strategies to address the challenges in rare disease drug development (see the accompanying paper), the Real-World Evidence (RWE) Scientific Working Group of the American Statistical Association Biopharmaceutical Section reviews the roles of RWD and RWE in clinical trials for drugs treating rare diseases. This paper summarizes relevant guidance documents and frameworks by selected regulatory agencies and the current practice on the use of RWD and RWE in natural history studies and the design, conduct, and analysis of rare disease clinical trials. A targeted learning roadmap for rare disease trials is described, followed by case studies on the use of RWD and RWE to support a natural history study and marketing applications in various settings. |
| format | Preprint |
| id |
arxiv_https___arxiv_org_abs_2410_06586 |
| institution | arXiv |
| publishDate | 2024 |
| record_format | arxiv |
| spellingShingle | Use of Real-World Data and Real-World Evidence in Rare Disease Drug Development: A Statistical Perspective Chen, Jie Gruber, Susan Lee, Hana Chu, Haitao Lee, Shiowjen Tian, Haijun Wang, Yan He, Weili Jemielita, Thomas Song, Yang Tamura, Roy Tian, Lu Zhao, Yihua Chen, Yong van der Laan, Mark Nie, Lei Applications Real-world data (RWD) and real-world evidence (RWE) have been increasingly used in medical product development and regulatory decision-making, especially for rare diseases. After outlining the challenges and possible strategies to address the challenges in rare disease drug development (see the accompanying paper), the Real-World Evidence (RWE) Scientific Working Group of the American Statistical Association Biopharmaceutical Section reviews the roles of RWD and RWE in clinical trials for drugs treating rare diseases. This paper summarizes relevant guidance documents and frameworks by selected regulatory agencies and the current practice on the use of RWD and RWE in natural history studies and the design, conduct, and analysis of rare disease clinical trials. A targeted learning roadmap for rare disease trials is described, followed by case studies on the use of RWD and RWE to support a natural history study and marketing applications in various settings. |
| title | Use of Real-World Data and Real-World Evidence in Rare Disease Drug Development: A Statistical Perspective |
| topic | Applications |
| url | https://arxiv.org/abs/2410.06586 |